Latest news

• Orchard surpasses IPO expectations, raising $200m

  Orchard gained ex-vivo gene therapy Strimvelis, a treatment for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), from the deal, along with a clinical programme for beta thalassaemia, which

• Celgene to launch five new products through to 2020

  myelofibrosis, thalassaemia therapy luspatercept, and CAR-T candidates lisocabtagene maraleucel (liso-cel) for lymphoma and bb2121 for multiple myeloma. ... read-outs from the MEDALIST trial of luspatercept in myelodysplastic syndrome (MDS) and the

• Orchard plans IPO as gene therapy pipeline advances

  beta thalassaemia – which has just bagged a Priority Medicines (PRIME) designation from the EMA. ... It has been tested so far in nine patients with transfusion-dependent beta-thalassemia, the most severe form of the disease.

• GSK hands rare disease assets over to Orchard

  Along with Strimvelis, GSK is passing over two therapies in late-stage clinical development for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical programme for beta thalassaemia.

• Novo Nordisk pays $400m for EpiDestiny’s sickle cell drug

  Novo Nordisk has bolstered its biopharma unit with a global licensing deal for a small-molecule drug for genetic blood disorders sickle cell disease and beta thalassaemia. ... And as an oral, once-daily drug EpiDestiny’s candidate could be easier to